Cereno comments on EU orphan designation for CS1
Cereno Scientific has received Orphan Medicinal Product Designation in the EU for CS1 in Pulmonary Arterial Hypertension, a rare cardiovascular disease. The decision comes as the company awaits topline data from a phase II trial evaluating CS1 in PAH. BioStock spoke with Cereno’s CEO Sten R. Sörensen to get his take on why this is a key milestone for the company.
Cereno Scientific is a clinical stage biotech company developing treatments for cardiovascular diseases (CVDs). The ultimate goal is to improve the quality of life and prolong life for people suffering from rare and common CVDs, and the work done so far suggests that the goal is within reach.
CS1 – unleashing the potential of epigenetic modulation in PAH
The company uses novel approaches for its drug development, with a focus on delivering disease-modifying therapies. Lead asset CS1, an innovative reformulation of the well-established anti-epileptic drug valproic acid (VPA), is a histone deacetylase inhibitor (HDACi) – an epigenetic modulator – with the potential to restructure (reverse-remodel) the blood vessels, ameliorate fibrosis, inflammation and thrombosis and ultimately lower the pulmonary arterial pressure. These properties make CS1 stand out against conventional PAH treatments, which do not address the core cause of the disease.
Read more about how Cereno is driving a paradigm shift in this field thanks to this novel therapeutic approach here.
Due to CS1’s potential to address multiple pathogenic mechanisms of PAH, Cereno has chosen to focus on this disease. PAH is a rare and devastating CVD causing an abnormal rise in pulmonary arterial blood pressure due primarily to vascular changes in the blood vessels transporting deoxygenated blood from the heart to the lungs. Consequently, the right side of the heart has to work excessively, eventually leading to heart failure and death within just a few years. CS1 has the potential to be an effective, safe and disease-modifying drug and the aim of CS1’s development is to offer improved quality of life and prolonged life for patients with PAH.
Orphan drug designation for CS1
The rare nature of PAH, combined with the significant unmet medical need for this disease, means that it qualifies as an orphan disease from a regulatory standpoint. Cereno was granted Orphan Drug Designation (ODD) for CS1 by the FDA in 2020. This gave the company a range of regulatory benefits that could result in a faster and simpler path to market approval and launch in the US.
Now, Cereno can enjoy similar benefits across the 27 EU countries, as CS1 was recently granted Orphan Medicinal Product Designation (OMPD) by the European Commission, following a recommendation from the European Medicines Agency (EMA).The main advantage of the provision is a 10-year market exclusivity period post-authorization – similar to the ODD, which offers a 7-year market exclusivity period in the US.
This regulatory milestone and subsequent market exclusivity complement the patent protection for CS1 in the EU and solidifies the business case for CS1 in Europe.
Phase II trial with CS1
The news comes as Cereno continues its evaluation of CS1’s safety, tolerability, pharmacokinetics and exploratory efficacy in a phase II trial in PAH. The study will also provide insights for planning subsequent trials of CS1.
Patient recruitment to the phase II trial was closed in July, based on a recommendation from the Study Clinical Steering Committee, and topline data are expected in Q3.
On January 30th, 2024, CS1 was approved by the FDA for Expanded Access, or “Compassionate Use,” giving patients who have completed the trial the opportunity to continue taking the drug if deemed beneficial by the investigators and patients. Just a few days ago, the first patient was dosed within the Expanded Access Program (EAP), and Cereno looks forward to seeing more patients enrolled in this program, to benefit from continued CS1 treatment.
Read more about how the EAP strengthens the case for CS1 here, and don’t miss this BioStock interview with Cereno’s CEO Sten R. Sörensen where he talks in depth about the benefits of the EAP.
CEO insights
BioStock caught up with Cereno’s CEO Sten R. Sörensen to learn about the OMPD benefits, how the designation will impact CS1’s continued development and the commercial opportunities this opens up for the company.
Sten, how does Orphan Medicinal Product Designation benefit the development process for CS1?
– The OMPD offers Cereno significant advantages, including a 10-year market exclusivity for CS1 post-authorization and various benefits during the development process such as fee reductions for market authorization applications, scientific advice and protocol assistance, accelerated assessment as well as assistance with regulatory procedures. The mentioned orphan incentives will all togheter facilitiate our efforts to bring CS1 to patients with PAH in all 27 EU countries in a timely manner.
How does the EU OMPD complement the ODD already granted by the FDA?
– The EU and the US are both large pharmaceutical markets, both in terms of number of patients and total market value. We are delighted that CS1 now holds enhanced protection, via market exlusitivity when approved, through the ODD and OMPD in the US as well as in Europe. The market exclusivity complements our patents in these markets.
Looking at the market potential within PAH, what commercial opportunities does this latest regulatory milestone open up for Cereno?
– Together, the ODD and OMPD provides a coordinated framework of incentives and regulatory support that, combined with the strong, and growing, patent protection for CS1 in these markets, lay a solid ground for market outreach once CS1 has received marketing authorization. The OMPD also secures access to a centralized authorization procedure, one application for all EU countries, yielding a less cumbersome road to approval and for potentially earlier returns on investment for the Company as well as our shareholders.
Recently, you released the news that the first patient was dosed in the Expanded Access Program, what is the significance of this?
– The EAP provides value both to patients suffering from PAH as well as for Cereno. Under the EAP, patients that have completed the Phase II trial have the opportunity to, after being judged suitable and to benefit from CS1 treatment by investigators, continue CS1 treatment of PAH when no comparable or satisfactory alternative therapy options are available. The EAP also allows Cereno to, under a formal FDA-approved protocol, collect safety and efficacy data from long-term exposure to CS1 in patients with PAH, to be utilized in regulatory discussions and Phase IIb/III pivotal study design planning.
What are the next steps the phase II trial of CS1 in PAH that we should most be looking forward to?
– Our next major milestone is the release of the topline results from the phase II trial. Based on a recommendation from the Study Clinical Steering Committee, we decided to close patient recruitment to study CS1-003 by July 1st. The Study Clinical Steering Committee concluded that there is sufficient data for evaluating the next steps in development. Topline results will be shared during Q3.
The content of BioStock’s news and analyses is independent but the work of BioStock is to a certain degree financed by life science companies. The above article concerns a company from which BioStock has received financing.
